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Int. J. Mol. Sci. 2016, 17(7), 1000; doi:10.3390/ijms17071000

Site-Specific Genome Engineering in Human Pluripotent Stem Cells

1,2,3
and
1,2,3,*
1
Leibniz Research Laboratories for Biotechnology and Artificial Organs (LEBAO), Department of Cardiothoracic, Transplantation and Vascular Surgery, Hannover Medical School, 30625 Hannover, Germany
2
Regenerative Biology and Reconstructive Therapies (REBIRTH), Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany
3
Biomedical Research in Endstage and Obstructive Lung Disease (BREATH), Member of the German Center for Lung Research (DZL), Hannover Medical School, 30625 Hannover, Germany
*
Author to whom correspondence should be addressed.
Academic Editor: Izuho Hatada
Received: 3 May 2016 / Revised: 16 June 2016 / Accepted: 20 June 2016 / Published: 24 June 2016
(This article belongs to the Special Issue Genome Editing)
View Full-Text   |   Download PDF [862 KB, uploaded 24 June 2016]   |  

Abstract

The possibility to generate patient-specific induced pluripotent stem cells (iPSCs) offers an unprecedented potential of applications in clinical therapy and medical research. Human iPSCs and their differentiated derivatives are tools for diseases modelling, drug discovery, safety pharmacology, and toxicology. Moreover, they allow for the engineering of bioartificial tissue and are promising candidates for cellular therapies. For many of these applications, the ability to genetically modify pluripotent stem cells (PSCs) is indispensable, but efficient site-specific and safe technologies for genetic engineering of PSCs were developed only recently. By now, customized engineered nucleases provide excellent tools for targeted genome editing, opening new perspectives for biomedical research and cellular therapies. View Full-Text
Keywords: targeted genome engineering; human iPSCs; zinc-finger nucleases (ZFNs); transcription activator-like effector nuclease (TALEN); clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 targeted genome engineering; human iPSCs; zinc-finger nucleases (ZFNs); transcription activator-like effector nuclease (TALEN); clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Merkert, S.; Martin, U. Site-Specific Genome Engineering in Human Pluripotent Stem Cells. Int. J. Mol. Sci. 2016, 17, 1000.

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